EU/3/10/799: Orphan designation for the treatment of cowpox infection

Cowpox is a viral infection that is most commonly transmitted to humans by infected domestic cats. Symptoms of human infection are malaise (feeling unwell) and painful fluid-filled blisters usually on the hands.

Cowpox infection can be debilitating as some patients develop widespread skin lesions or a lesion in the eye. The infection may be life threatening in patients with a weakened immune system (the body's natural defences).

At the time of designation, cowpox infection affected less than 0.001 in 10,000 people in the EU*. This is equivalent to a total of fewer than 50 people and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 506,500,000 (Eurostat 2010).

At the time of designation, no satisfactory methods of treatment were authorised in the EU for cowpox infection.

Tecovirimat is expected to work by interfering with a protein normally found on the surface of the cowpox virus particle. As a result, the virus cannot reproduce normally, slowing down the spread of infection.

The effects of tecovirimat have been evaluated in experimental models.

At the time of submission of the application for orphan designation, four clinical trials with tecovirimat in healthy volunteers had been completed.

At the time of submission, tecovirimat was not authorised anywhere in the EU for the treatment of cowpox infection or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 July 2010 recommending the granting of this designation.

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.